The Orphan Drug Act at 40: Legislative Triumph and the Challenges of Success.
Milbank Q
; 102(1): 83-96, 2024 Mar.
Article
en En
| MEDLINE
| ID: mdl-38087888
ABSTRACT
Policy Points The Orphan Drug Act (ODA) was the result of patient advocacy and by many measures has been strikingly successful. However, approximately 95% of the more than 7,000 known rare diseases still have no US Food and Drug Administration-approved treatment. The ODA's success led to sustained criticism of high drug prices, often for products that have orphan drug indications. Critics misconstrue the ODA's intent and propose reducing its incentives instead of pursuing policies focused on addressing broader prescription drug price challenges that exist in both the orphan and nonorphan drug market. Patients and their families will continue to defend the purpose and integrity of the ODA and to drive investments into rare disease research and clinical development.
Palabras clave
Texto completo:
1
Colección:
01-internacional
Banco de datos:
MEDLINE
Asunto principal:
Producción de Medicamentos sin Interés Comercial
/
Aprobación de Drogas
Límite:
Humans
País/Región como asunto:
America do norte
Idioma:
En
Revista:
Milbank Q
Asunto de la revista:
MEDICINA SOCIAL
/
SAUDE PUBLICA
/
SERVICOS DE SAUDE
Año:
2024
Tipo del documento:
Article